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Losmapimod

Facioscapulohumeral Muscular Dystrophy (FSHD)

Phase 3Active (Top-line data reported)NCT05397470

Key Facts

Indication
Facioscapulohumeral Muscular Dystrophy (FSHD)
Phase
Phase 3
Status
Active (Top-line data reported)
Company

About Fulcrum Therapeutics

Fulcrum Therapeutics is a clinical-stage biotech company with a mission to develop small molecule therapies that treat the root cause of genetically defined rare diseases by modulating gene expression. Its core achievement is the development of a proprietary functional genomics platform that identifies master regulator proteins to create targeted treatments. The company's strategy focuses on high-unmet-need disorders with clear genetic drivers, such as FSHD and sickle cell disease, leveraging regulatory pathways for rare diseases. Fulcrum's lead asset, losmapimod for FSHD, has completed Phase 3 trials, positioning the company for a potential near-term regulatory filing and commercialization.

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Other Facioscapulohumeral Muscular Dystrophy (FSHD) Drugs

DrugCompanyPhase
DX5057Altay TherapeuticsPreclinical
Restem-LRestemUnknown
AOC 1020Avidity BiosciencesPhase 1/2
DYNE-301Dyne TherapeuticsPhase 1/2